On May 9, 2017 Foamix Pharmaceuticals Ltd. (NASDAQ: FOMX) ("Foamix Pharmaceuticals" or the "Company"), a clinical stage specialty pharmaceutical company focused on developing and commercializing proprietary topical foams to address unmet needs in dermatology, reported financial results for the three months ended March 31, 2017 (Press release, Foamix, MAY 9, 2017, View Source [SID1234518982]).

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Clinical, business and corporate developments for the three months ended March 31, 2017 and to date:
On March 27, 2017, we provided the top-line data from our two Phase 3 clinical trials (Trial 04 and 05) for FMX101 in the treatment of moderate-to-severe acne. In the intent-to-treat analysis, FMX101 demonstrated statistical significance compared to vehicle on both co-primary endpoints in Trial 05 (specifically the absolute reduction in inflammatory lesions at week 12, and investigator global assessment (IGA) treatment success at week 12 compared to baseline). In Trial 04, statistical significance was demonstrated for FMX101 compared to vehicle in the co-primary endpoint of absolute reduction in inflammatory lesions, however, statistical significance was not achieved in the co-primary endpoint of IGA treatment success.

On May 3, 2017, we provided new data from our two Phase 3 clinical trials for FMX101, including pooled analysis of our co-primary endpoints and certain secondary clinical endpoints (absolute reduction of non-inflammatory lesions at week 12; and percent change in inflammatory lesions at weeks 3, 6, 9 and 12). Highlights from our further analyses included:
Statistical significance was demonstrated for FMX101 compared to vehicle in the pooled analysis of both co-primary endpoints – absolute reduction of inflammatory lesions and Investigator’s Global Assessment (IGA)
% Change in inflammatory lesion count was statistically significant in both Trials 04 and 05 at all timepoints (beginning at Week 3)
Non-inflammatory lesion count reduction at Week 12 was statistically significant in both Trials 04 and 05
Overall high level of patient satisfaction with FMX101 (based on patient satisfaction questionnaires).
FMX101 was generally safe and tolerable. No serious adverse events drug-related systemic side effects were recorded.

Further to sharing the detailed analyses, we announced that based on the results of the first two pivotal trials (Trial 04 and 05), we intend to conduct a third U.S. Phase 3 trial in patients with moderate-to-severe acne. This double-blind, vehicle-controlled trial is planned to enroll 1,500 patients who will be randomized 1:1 (FMX101 vs vehicle) across an estimated 80 investigator sites. The trial is expected to commence mid-year. If the results are positive, this trial will form the basis for a New Drug Application (NDA) which the company plans to submit in the second half of 2018.

The two Phase 3 clinical trials for FMX103 in patients with moderate-to-severe papulopustular rosacea are expected to commence mid-2017. We also announced on May 3, 2017, that we plan to increase the sample size for each of the two Phase 3 trials from 600 to 750 patients (total of 1,500 patients) randomized 2:1 (FMX103 vs vehicle) across an estimated 80 investigator sites in the U.S. FMX103 demonstrated clinically and statistically significant efficacy in treating moderate-to-severe rosacea in a Phase 2 trial which enrolled 233 patients across 18 sites in Germany.
During the first quarter of 2017 we successfully manufactured three registration-quality batches for FMX101.
U.S. Sales of Finacea Foam, azelaic acid 15% for the treatment of rosacea, continue to grow.

Based on sales of Finacea Foam reported by Bayer HealthCare AG for Q1, 2017 Foamix is entitled to royalty payments of $927,000, up 26% from the fourth quarter of 2016.

Finacea Foam was developed through a research and development collaboration between Foamix and Bayer, utilizing Foamix’s proprietary foam technology platform. The drug was launched by Bayer in the USA in September 2015.

Financial highlights for the three months ended March 31, 2017:
Total revenues were $927,000 compared with $745,000 for the three months ended March 31, 2016. The increase is due to increase in sales of Finacea Foam by Bayer HealthCare AG.

Research and development expenses were $12.7 million, compared with $3.6 million in the three months ended March 31, 2016. This increase resulted primarily from an increase in costs relating to the FMX101 and FMX103 clinical trials as well as an increase in payroll and related expenses due to an increase in the number of R&D employees.

Selling, general and administrative expenses were $2.8 million, compared with $1.7 million in the three months ended March 31, 2016. The increase in selling, general and administrative expenses resulted primarily from increases in payroll and other payroll-related expenses, market research costs, advisors, maintenance and office expenses.

Operating expenses totaled $15.5 million, compared with $5.3 million in the three months ended March 31, 2016.
Net loss was $14.4 million or $0.39 per share, basic and diluted, compared with a loss of $4.5 million or $0.15 per share, basic and diluted, for the three months ended March 31, 2016.

Cash and investments as of March 31, 2017 totaled $118.7 million, compared with $131.0 million as of December 31, 2016.

Management overview
On March 27, 2017, we provided the top-line data from our two Phase 3 clinical trials (Trial 04 and 05) for FMX101 in the treatment of moderate-to-severe acne. In the intent-to-treat analysis, FMX101 demonstrated statistical significance compared to vehicle on both co-primary endpoints in Trial 05 (specifically the absolute reduction in inflammatory lesions at week 12, and investigator global assessment (IGA) treatment success at week 12 compared to baseline). In Trial 04, statistical significance was demonstrated for FMX101 compared to vehicle in the co-primary endpoint of absolute reduction in inflammatory lesions, however, statistical significance was not achieved in the co-primary endpoint of IGA treatment success. On May 3, 2017, we provided new data from our two Phase 3 clinical trials for FMX101, including pooled analysis of our co-primary endpoints and certain secondary clinical endpoints (absolute reduction of non-inflammatory lesions at week 12; and percent change in inflammatory lesions at weeks 3, 6, 9 and 12). Statistical significance was demonstrated for FMX101 compared to vehicle in the pooled analysis of the co-primary endpoints as well as the secondary endpoints presented.

Co-primary endpoint – Absolute change from baseline in inflammatory lesion count at week 12:
Trial 04: reduction of 14.16 lesions (or -14.16) for FMX101 and reduction of 11.17 lesions (or -11.17) for the vehicle (p<0.01)

Trial 05: -13.46 for FMX101 and -10.72 for vehicle (p<0.01)
Pooled Analysis: Absolute change in inflammatory lesion count was -13.79 for the FMX101, 4% treatment group and -10.94 for vehicle (p=0.0001)

Co-primary endpoint – Proportion of patients with Investigator’s Global Assessment (IGA) success at week 12:
Trial 04: IGA treatment success for FMX101, 4% treatment group was 8.09% versus 4.77% in vehicle (p=0.2178)
Trial 05: IGA treatment success for FMX101, 4% treatment group was 14.67% versus 7.89% in vehicle (p<0.05)
Pooled Analysis: IGA treatment success was 11.51% for FMX101, 4% treatment group and 6.34% for vehicle (p<0.05)

Secondary efficacy endpoint – Percent change from baseline in inflammatory lesion count at weeks 3, 6, 9 and 12:
Trial 04: reduction of 29% for FMX101 vs. reduction of 19% for vehicle, or -29% vs. -19%, at week 3 (p<.001); -37% vs. -26% at week 6 (p<.001); -42% vs. -28% at week 9 (p<.0001); and -44% vs. -34% at week 12 (p<0.01)

Trial 05: reduction of 34% for FMX101 vs. reduction of 21% for vehicle, or -34% vs. -21%, at week 3 (p<.0001); -39% vs. -27% at week 6 (p<.0001); -43% vs. -31% at week 9 (p<.001); and: -43% vs. -34% at week 12 (p<0.01)

Secondary efficacy endpoint – Absolute change from baseline in non-inflammatory lesion count at week 12:
Trial 04: reduction of 16.45 lesions (or -16.45) for the FMX101, 4% treatment group and reduction of 10.30 lesions (or -10.30) for the vehicle (p<0.01)

Trial 05: reduction of 13.20 (or -13.20) for the FMX101, 4% treatment group and reduction of 7.00 (or -7.00) for the vehicle (p<0.05)

Pooled Analysis: Absolute change in non-inflammatory lesion count was -14.76 for the FMX101, 4% treatment group and -8.64 for vehicle (p<0.01)

As we announced on May 3, 2017, based on the results of the first two pivotal trials (Studies 04 and 05), the company intends to conduct a third U.S. Phase 3 trial in patients with moderate-to-severe acne. If the results are positive, this trial will form the basis for an NDA which the company plans to submit in the second half of 2018. This planned clinical trial will be conducted at approximately 80 investigator sites in the U.S. In order to achieve the necessary statistical power compared with the prior Phase 3 trials, the target patient enrollment number has been increased to 1,500. Patients will be randomized 1:1 to receive either FMX101 (minocycline foam 4%) or vehicle foam once daily over 12 weeks. The co-primary efficacy endpoints will be identical to the prior Phase 3 trials: (1) mean change from baseline in the inflammatory lesion count, and (2) proportion of patients with IGA scores of "Clear" or "Almost Clear", with improvement of at least two grades from baseline. The inclusion criteria will be consistent with the prior Phase 3 trials.

We intend to meet with the FDA to review the results of our Phase 3 clinical trials for FMX101 (Trial 04 and 05) and our third Phase 3 trial, which we expect to commence mid-year.

Following the results of the first two pivotal trials for FMX101 in moderate-to-severe acne, we have also reviewed our Phase 3 program for FMX103 in papulopustular rosacea, which is expected to commence around mid-2017. Based on the outcome of the Phase 3 studies for FMX101, and the planned increase in the number of patients to be enrolled in the third Phase 3 trial in acne, we also intend to increase the sample size for the two planned Phase 3 studies for FMX103 in papulopustular rosacea. The sample size will be increased from 600 patients per trial to 750 patients per trial, for a total of 1,500 patients across the two studies.

Regarding manufacturing, we have successfully completed the scale-up process for FMX101 to a commercial batch size of one-ton. The production of three registration batches has been completed.

In addition to our internal drug development pipeline, we have development and license agreements relating to our proprietary foam technology with other pharmaceutical companies, including Bayer Healthcare and others, in various stages of development and commercialization. Our agreements with these licensees entitle us to development fees, contingent payments and royalties upon commercialization.

In September 2015, Bayer Healthcare began selling Finacea Foam (azelaic acid 15% for the treatment of rosacea) in the U.S. Finacea foam is a prescription foam product which was developed as part of a research and development collaboration between Foamix and Bayer, utilizing Foamix’s proprietary foam technology platform.

According to our license agreement with Bayer, we are entitled to royalties upon commercialization of Finacea Foam.

For the three months ended March 31, 2017, we were entitled to royalties from Bayer in an amount of $927,000, up 26% from the fourth quarter of 2016.

The Company is currently well-capitalized and has sufficient cash to fund our key development programs (FMX101 and FMX103) through NDA registration.
Financial results for the three months ended March 31, 2017

Revenues
Total revenues for the three-month ended March 31, 2017 were $927,000 compared with $745,000 for the three months ended March 31, 2016. The increase is due to increase in sales of Finacea Foam by Bayer HealthCare AG.

Operating Expenses
Our operating expenses for the three months ended March 31, 2017, and three months ended March 31, 2016, were as follows:

Research and Development Expenses
Research and development expenses increased by $9.1 million, or 255%, from $3.6 million in the three months ended March 31, 2016, to $12.7 million in the three months ended March 31, 2017. The increase in research and development expenses resulted primarily from an increase of $7.9 million in costs relating to the FMX101 and FMX103 clinical trials and an increase of $1.1 million in payroll and payroll related expenses (including bonuses and equity-based compensation) due to an increase in the number of R&D employees.

Selling, General and Administrative Expenses
Selling, general and administrative expenses increased by $1.1 million, or 65%, from $1.7 million in the three months ended March 31, 2016, to $2.8 million in the three months ended March 31, 2017. The increase in selling, general and administrative expenses resulted primarily from an increase of $300,000 in payroll and other payroll-related expenses (including bonuses and equity-based compensation), an increase of $250,000 in advisors, consultants and other professional services, $102,000 in market research costs and $122,000 in rent, maintenance and office expenses.

Finance Income, Net
For the three months ended March 31, 2017, we recorded financial income of $257,000 compared to financial income of $174,000 recorded for the three months ended March 31, 2016. The financial income for the three months ended March 31, 2017 and 2016 resulted mostly from interest and financial gains from our cash investments.

Net Loss
For the three months ended March 31, 2017, we recorded a loss of $14.4 million or $0.39 per share, basic and diluted, compared with a loss of $4.5 million or $0.15 per share, basic and diluted, for the three months ended March 31, 2016.

Liquidity and Capital Resources
As of March 31, 2017, we had cash and investments of $118.7 million, compared with $131.0 million as of December 31, 2016. The decrease was mostly due to operating expenses primarily relating to the clinical trials. During the three months ended March 31, 2017 we used $12.1 million in cash in our operations compared to $7.0 million used in operating activities in the three months ended March 31, 2016.

On May 10, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the U.S. Food and Drug Administration (FDA) has authorized the Company’s investigational new drug (IND) application for FATE-NK100 in advanced solid tumors (Press release, Fate Therapeutics, MAY 9, 2017, View Source [SID1234518971]). The Company plans to promptly initiate the DIMENSION study, an open-label, multi-center, accelerated dose-escalation clinical trial of FATE-NK100 as a monotherapy and in combination with monoclonal antibody therapy in subjects who have failed approved therapies.

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"The FDA’s clearance of this IND is a significant milestone, ushering in the opportunity to develop a powerful new immunologic approach to solid tumors that bridges innate and adaptive immunity. Activated NK cells can intrinsically seek out and directly kill transformed cancer cells, including antibody-coated tumor cells, and can trigger a long-lived adaptive T-cell immune response through pro-inflammatory cytokine release," said Chris Storgard, M.D., Chief Medical Officer of Fate Therapeutics. "We are excited to begin this clinical investigation of FATE-NK100, which has demonstrated in preclinical studies the potential to selectively eliminate tumor cells while leaving normal healthy cells unharmed."

The Company plans to enroll subjects in the DIMENSION study across three FATE-NK100 treatment arms in an outpatient setting: as monotherapy for solid tumor malignancies, including small cell lung cancer and hepatocellular carcinoma; in combination with trastuzumab for advanced HER2+ cancers, including breast and gastric cancers; and in combination with cetuximab for advanced EGFR1+ cancers, including colorectal and head and neck cancers. Activation of a patient’s NK cells has been clinically proven to play a major role in the anti-tumor efficacy of many monoclonal antibodies, including trastuzumab and cetuximab.

In preclinical models, FATE-NK100 has been shown to significantly augment antibody-directed cellular cytotoxicity against cancer cells when administered in combination with a monoclonal antibody, including antibodies that target CD20, HER2 and EGFR antigens. Additionally, FATE-NK100 has displayed enhanced anti-tumor activity across a broad range of hematologic and solid tumors, improved persistence and increased resistance to immune checkpoint pathways in preclinical studies compared to NK cell therapies that are being clinically administered today.

The primary objective of the DIMENSION study is to evaluate the safety and determine the maximum tolerated dose of a single intravenous infusion of FATE-NK100. Other objectives include determination of objective response rate, time-to-tumor progression, progression-free survival and overall survival.

Each of the three arms of the DIMENSION study will enroll in parallel utilizing accelerated dose-escalation, with each arm expected to include an expansion cohort of up to an additional twenty subjects at the maximum tolerated dose level. In addition, observation of a RECIST partial response or greater will enable additional expansion of up to ten subjects in that tumor type.

About FATE-NK100

FATE-NK100 is a first-in-class natural killer (NK) cell cancer immunotherapy comprised of adaptive memory NK cells, a highly specialized and functionally distinct subset of activated NK cells expressing the memory-like activating receptor NKG2C and the maturation marker CD57. FATE-NK100 is produced through a feeder-free, seven-day manufacturing process during which NK cells sourced from a healthy donor are activated ex vivo with pharmacologic modulators. An investigator-initiated clinical trial of FATE-NK100 is currently being conducted at the Masonic Cancer Center, University of Minnesota for the treatment of refractory or relapsed acute myelogenous leukemia.

On May 9, 2017 Nektar Therapeutics (Nasdaq: NKTR) reported its financial results for the first quarter ended March 31, 2017 (Press release, Nektar Therapeutics, MAY 9, 2017, View Source [SID1234518967]).

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Cash and investments in marketable securities at March 31, 2017 were $362.0 million as compared to $389.1 million at December 31, 2016.

"I am very pleased with our continued success in advancing the Nektar pipeline, driven by our expanding research in immuno-oncology and immunology that continues to generate highly valuable new clinical candidates," said Howard W. Robin, President and Chief Executive Officer of Nektar. "In March, we announced overwhelmingly positive efficacy and safety results from our Phase 3 study of NKTR-181 in patients with chronic low back pain. Our Phase 1/2 study evaluating NKTR-214 as a combination regimen with Opdivo in collaboration with Bristol-Myers Squibb is advancing and we look forward to reporting initial data from the first patients in this trial at ASCO (Free ASCO Whitepaper). In Q1, we also initiated a first-in-human trial for NKTR-358, our proprietary Treg stimulator, which has the potential to become a first-in-class resolution therapeutic for a wide range of immune-mediated disorders. We plan to report the results from this trial at a medical meeting in the second half of 2017."

Revenue for the first quarter of 2017 was $24.7 million as compared to $58.9 million in the first quarter of 2016. Revenue in the first quarter of 2016 was higher primarily because of the recognition of $28.0 million received from AstraZeneca for the sublicense of MOVENTIG to Kirin in Europe. In addition, product sales were $4.8 million in the first quarter of 2017 as compared to $14.1 million in the first quarter of 2016.

Total operating costs and expenses for the first quarter of 2017 were $79.2 million as compared to $68.4 million in the first quarter of 2016. Total operating costs and expenses increased primarily as a result of higher research and development (R&D) expense in the first quarter of 2017. R&D expense in the first quarter of 2017 was $61.1 million as compared to $49.3 million for the first quarter of 2016 and was higher in the first quarter of 2017 primarily due to expenses for our NKTR-214 and NKTR-358 programs.

General and administrative expense was $12.0 million in the first quarter of 2017 as compared to $10.2 million in the first quarter of 2016.

In the first quarter of 2017, net loss was $63.9 million, or $0.42 loss per share as compared to net loss of $19.5 million, or $0.14 loss per share in the first quarter of 2016. The loss was higher year over year primarily because of the recognition of $28.0 million received from AstraZeneca for the sublicense of MOVENTIG to Kirin in Europe in the first quarter of 2016.

The company also announced upcoming presentations at the following scientific congresses during the second quarter of 2017:

Oxford Global 2nd Annual Advances in Immuno-Oncology Congress, London, UK

Oral Presentation: "NKTR-255: Accessing The Immunotherapeutic Potential of IL-15"
Presenter: Jonathan Zalevsky, Ph.D.
Session: Pre-clinical Immuno-Oncology
Date and Time: May 15, 2017 – 2:20 p.m. – 2:50 p.m. BST

SMI 16th Annual Pain Therapeutics Conference, London, UK

Key Note Address: "NKTR-181: Separating Analgesia from Euphoria in a Novel Opioid Agonist for Chronic Pain"
Presenter: Stephen Doberstein, Ph.D.
Session: Opioid Addiction
Date and Time: May 23, 2017 – 9:50 a.m. – 10:30 a.m. BST

2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, Chicago, IL

Abstract 2545/Poster 37: "Effect of a novel IL-2 cytokine immune agonist (NKTR-214) on proliferating CD8+T cells and PD-1 expression on immune cells in the tumor microenvironment in patients with prior checkpoint therapy." Bernatchez, C., et al.
Poster Session: Developmental Therapeutics—Clinical Pharmacology and Experimental Therapeutics
Date and Time: June 5, 2017 – 8:00 a.m. – 11:30 a.m. CDT
Location: Hall A

Abstract TPS1120/Poster 105a: "ATTAIN: Phase 3 study of etirinotecan pegol (EP) vs treatment of physician’s choice (TPC) in patients (pts) with metastatic breast cancer (MBC) who have stable brain metastases (BM) previously treated with an anthracycline, a taxane, and capecitabine (ATC)." Tripathy, D., et al.
Poster session: Breast Cancer – Metastatic
Date and Time: June 4, 2017 – 8:00 a.m. – 11:30 a.m. CDT
Location: Hall A

Abstract e14040: "A phase 1/2 study of a novel IL-2 cytokine, NKTR-214, and nivolumab in patients with select locally advanced or metastatic solid tumors." Diab, A., et al.
Publication abstract to be included online in the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting Proceedings, a Journal of Clinical Oncology supplement.

2017 International Conference on Opioids (ICOO 2017), Boston, MA

Poster 31: "NKTR-181 Produces Full CNS µ-Opioid Agonism With Significantly Lower Abuse Potential": Odinecs, A., et al.
Poster session: Session 2
Date and Time: Monday, June 12, 2017 – 8:00 a.m. – 6:00 p.m. EDT

Inaugural Immuno-Oncology Targets Conference, Boston, MA

Oral Presentation: "NKTR-214 Plus NKTR-262, a Scientifically-Guided Rational Combination Approach for Immune Oncology"
Presenter: Jonathan Zalevsky, Ph.D.
Session: Rational Combination Immunotherapy
Date and Time: June 15, 2017 – 12:00 p.m. EDT

Nektar Analyst & Investor Event at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting, Chicago, IL

Nektar will host an analyst and investor event with clinical investigators during the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Meeting in Chicago. The program will include a presentation and discussion of updated clinical data for the company’s CD122-biased agonist, NKTR-214. Data from two studies of NKTR-214 will be reviewed at the event, including the Phase 1 dose-escalation study of NKTR-214 in combination with nivolumab in patients with melanoma, renal cell carcinoma and non-small cell lung cancer (PIVOT-02); and the Phase 1 study of monotherapy NKTR-214 in patients with advanced solid tumors (EXCEL).

Presenters will include Dr. Adi Diab, Assistant Professor, Melanoma Medical Oncology at the University of Texas MD Anderson Cancer Center, Dr. Nizar Tannir, Professor, Genitourinary Medical Oncology at the University of Texas MD Anderson Cancer Center and Dr. Michael Hurwitz, Assistant Professor of Medicine (Medical Oncology) at Yale Cancer Center.

Date and Time: June 3, 2017 – 6:00 p.m. CDT
Webcast Link: View Source

On May 9, 2017 Navidea Biopharmaceuticals, Inc. (NYSE MKT: NAVB) ("Navidea" or "the Company"), a company focused on the development and commercialization of precision immunodiagnostic agents, reported its financial results for the first quarter of 2017 (Press release, Navidea Biopharmaceuticals, MAY 9, 2017, View Source [SID1234518966]).

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Navidea reported total revenue (excluding discontinued operations) for the quarter of $580,000. Net income attributable to common stockholders was $85.6 million.

"Navidea ended the first quarter with strong momentum built upon the strategic plan developed over the past two quarters. Our strategy is designed to maximize the value of our proprietary macrophage-targeting technology by developing and out-licensing promising imaging and therapeutic products," said Michael Goldberg, M.D., Navidea’s President and CEO. Dr. Goldberg continued, "Our completed sale of the North American rights to Lymphoseek to Cardinal Health 414, LLC ensures that our focus remains on product development going forward. We are confident that our Manocept platform, properly developed, will yield both diagnostics and therapeutics that can generate significant value for our stockholders."

Product, Pipeline, and Business Updates

Lymphoseek

On March 3, 2017, Navidea completed the sale of the North American rights to Lymphoseek to Cardinal Health 414, receiving approximately $82 million at closing.
Navidea will have the opportunity to earn up to $227 million of additional consideration through 2026, with $17.1 million guaranteed over the next three years.
As a result of this closing, all liens on Navidea’s assets have been released, all frozen accounts have been transferred to Navidea’s control, and the majority of the loan from Platinum Partners has been repaid.
Manocept Immunodiagnostic Pipeline

The flexible and versatile Manocept platform acts as an engine for the design of targeted imaging molecules applicable to a range of diagnostic modalities, including single photon emission computed tomography ("SPECT"), positron emission tomography ("PET"), gamma-scanning (both imaging and topical) and intra-operative and/or optical-fluorescence detection. We have active clinical diagnostic programs in cardiovascular disease, rheumatoid arthritis, Kaposi’s sarcoma and colorectal cancer, diseases representing both major macrophage activation states.

Cardiovascular Disease – The results of a study to evaluate diagnostic imaging of emerging atherosclerosis plaque with Tc 99m tilmanocept were published in early release in the Journal of Infectious Diseases on January 16, 2017, confirming that the Tc 99m tilmanocept product can both quantitatively and qualitatively target non-calcified plaque in the aortic arch.

Colorectal Cancer and Synchronous Liver Metastases – During the first quarter of 2017, we initiated an imaging study in subjects with colorectal cancer and liver metastases via intravenous administration of Tc 99m tilmanocept.

Manocept Immunotherapeutic Development Pipeline (Macrophage Therapeutics)

Navidea’s majority-owned subsidiary, Macrophage Therapeutics, Inc. ("MT"), has developed processes for producing the first two therapeutic Manocept immunoconstructs consisting of a therapeutic molecule conjugated to moieties targeting CD206+ macrophages:

MT-1002, designed to specifically target and kill activated CD206+ macrophages by delivering doxorubicin; and
MT-2002, designed to inhibit the inflammatory activity of activated CD206+ macrophages by delivering a potent anti-inflammatory agent.
In the first quarter of 2017, MT completed its third vivo study dosing either MT-1002 or MT-2002 in a well-established mouse model of nonalcoholic fatty liver disease/nonalcoholic steatohepatitis and liver fibrosis, in which both compounds significantly reduced key disease parameters.

Also in the first quarter of 2017, we completed a series of predictive in vitro screening tests of the MT-1002 and MT-2002 therapeutic conjugates against the Zika and Dengue viruses and against Leishmaniosis. These evaluations were positive and MT will begin in vivo testing in the second or third quarter of 2017.

Financials

Our consolidated balance sheets and statements of operations have been reclassified, as required by current accounting standards, for all periods presented to reflect the line of business sold to Cardinal Health 414 as a discontinued operation. Accordingly, this discussion focuses on describing results of our operations as if we had not operated the discontinued operation during the periods being disclosed.

We recorded a net gain on the sale to Cardinal Health 414 of $88.7 million for the first quarter of 2017, including $16.5 in guaranteed consideration, which was discounted to the present value of future cash flows. The proceeds were offset by $3.3 million in estimated fair value of warrants issued to Cardinal Health 414, $2.0 million in legal and other fees related to the sale, $800,000 in net balance sheet dispositions and write-offs, and $4.6 million in estimated taxes.

Total revenues for the quarter ended March 31, 2017 were $580,000 compared to $948,000 in the first quarter of last year. Total operating expenses for the first quarter of 2017 were $3.7 million, compared to $4.7 million in the first quarter of last year. Research and development expenses for the first quarter of 2017 were $705,000, compared to $2.1 million in the first quarter of last year. The net decrease from 2016 to 2017 was primarily a result of decreases in NAV4694, Tc 99m tilmanocept and NAV5001 development costs, offset by increases in Manocept and therapeutics development costs, coupled with decreased compensation and related support costs. Selling, general, and administrative expenses for the first quarter of 2017 were $3.0 million, compared to $2.6 million in the first quarter of last year. The net increase was primarily due to increased legal and professional services, offset by decreased investor relations services, compensation and related support costs.

Navidea’s net income attributable to common stockholders for the quarter ended March 31, 2017 was $85.6 million, or $0.53 per share (basic), compared to a net loss of $3.7 million, or $0.02 per share, for the same period in 2016.

Navidea ended the quarter with $13.4 million in cash.

On May 9, 2017 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its first quarter 2017 financial results and provided an update on the company, including its progress with IPI-549, a potentially first-in-class immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase gamma (PI3K-gamma) (Press release, Infinity Pharmaceuticals, MAY 9, 2017, View Source [SID1234518964]).

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Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in a Phase 1 study in patients with advanced solid tumors. Dose-escalation data from the ongoing study were presented in April at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2017 in Washington, D.C. These data demonstrated that IPI-549 was well tolerated both as a monotherapy and in combination with Opdivo. Additionally, data from the monotherapy module of the study showed IPI-549 has a favorable pharmacokinetic (PK) and pharmacodynamic (PD) profile that supports once daily (QD) dosing. IPI-549 is believed to be the only selective PI3K-gamma inhibitor in clinical development.

"IPI-549 has a number of key attributes that have enabled us to rapidly enroll patients in our ongoing study. It is orally administered, is believed to be the only selective PI3K-gamma inhibitor in clinical development, and has demonstrated a favorable tolerability profile, both as a monotherapy as well as in combination with Opdivo," stated Adelene Perkins, Infinity’s chair and chief executive officer. "Additionally, there is a strong body of preclinical translational research supporting the program demonstrating that selectively targeting PI3K-gamma with IPI-549 reprograms macrophages from a pro-tumor to an anti-tumor phenotype and is able to help overcome resistance to checkpoint inhibition, representing a unique and potentially transformative approach within immuno-oncology."

"Our dose-escalation phases evaluating IPI-549 alone and with Opdivo are ongoing, and we are on track to initiate the expansion phases in the second half of the year. The expansion phases will allow us to generate additional clinical data from monotherapy treatment as well as from the combination of IPI-549 and Opdivo in specific types of solid tumors. We are pleased with the data we recently presented at the AACR (Free AACR Whitepaper) Annual Meeting 2017, and we expect to report additional data from this study in the second half of the year," continued Ms. Perkins.

The ongoing Phase 1 study is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 175 patients with advanced solid tumors. The study includes four phases, or modules: monotherapy dose escalation, combination therapy dose escalation, monotherapy expansion, and combination therapy expansion. The combination expansion module will include multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma, and squamous cell carcinoma of the head and neck (SCCHN) whose tumors show initial resistance or subsequently develop resistance to immune checkpoint blockade therapy. There is a great need for additional treatment options for the growing number of patients living with these cancers, which account for more than 17 percent of all new cancer cases in the U.S.1,2

Recent Developments

Dose-escalation modules of Phase 1 study of IPI-549 ongoing: Infinity reported today that it has completed an analysis of a cohort of patients who received IPI-549 40 mg QD and has begun enrolling patients in a cohort to evaluate IPI-549 60 mg QD. The company expects to complete the monotherapy dose-escalation module in the first half of the year and subsequently initiate patient enrollment in the monotherapy expansion module in the second half of 2017.

Infinity also reported today that it is continuing to evaluate data from the dose-escalation cohort evaluating IPI-549 30 mg QD in combination with Opdivo. The company expects to complete the combination dose-escalation module and initiate the combination expansion module in the second half of 2017.

Updated Phase 1 clinical data presented at AACR (Free AACR Whitepaper) Annual Meeting 2017: New Phase 1 data from a March 20, 2017, data cutoff were presented during a poster session at the AACR (Free AACR Whitepaper) Annual Meeting 2017. The presentation included data from 15 evaluable patients who received monotherapy doses of IPI-549 ranging from 10 mg to 40 mg QD and six evaluable patients who received IPI-549 20 mg QD in combination with Opdivo.

Data from the monotherapy dose-escalation module demonstrated that IPI-549 treatment was well tolerated. Among the 15 evaluable patients, no dose limiting toxicities or serious drug-related side effects occurred, and no side effects led to treatment discontinuation or dose reduction. The PK and PD properties of IPI-549 appeared favorable, with near-complete and sustained inhibition of PI3K-gamma at doses at or above 20 mg QD, supporting once daily dosing of IPI-549.

Preliminary data from the combination dose-escalation module evaluating 20 mg IPI-549 in combination with Opdivo demonstrated that the treatment regimen was well tolerated and did not result in new or unexpected side effects relative to the known safety profile of each treatment when administered as monotherapy. Among the six evaluable patients, no dose limiting toxicities or serious drug-related side effects occurred, and no side effects led to treatment discontinuation. The PK profile of IPI-549 in combination with Opdivo appeared favorable and suggested that Opdivo does not affect the PK properties of IPI-549.

Rationale for targeting PI3K-gamma and preclinical data for IPI-549 presented at AACR (Free AACR Whitepaper) Annual Meeting 2017: Jeffery Kutok, M.D., Ph.D., Infinity’s chief scientific officer, presented the rationale for targeting PI3K-gamma during an educational session at the AACR (Free AACR Whitepaper) Annual Meeting 2017. In contrast to the other three major PI3K isoforms, PI3K-gamma is highly expressed in tumor-associated macrophages and plays an important role in the pro-tumor function of these cells. Infinity’s scientists designed IPI-549 to be not only a very potent but also a highly selective PI3K-gamma inhibitor, and it has greater than 100-fold selectivity for PI3K-gamma over the other major PI3K isoforms.

During the educational session, Dr. Kutok also summarized the preclinical rationale for targeting PI3K-gamma previously reported in two publications in Nature.3,4 Preclinical research has demonstrated that blockade of PI3K-gamma signaling by treatment with IPI-549 results in a transcriptional reprogramming of macrophages. This reprogramming shifted macrophages in the tumor microenvironment from the M2, or pro-tumor phenotype, to the M1, or anti-tumor phenotype, increased the number and activity of anti-tumor T cells that attack the tumor and also increased the production of pro-inflammatory cytokines.

Preclinical data from multiple solid tumor models demonstrated that IPI-549 was active as a monotherapy and that IPI-549 administered in combination with checkpoint inhibition led to enhanced activity compared to either treatment alone. Additionally, preclinical data demonstrated that M2, pro-tumor macrophages are associated with resistance to checkpoint inhibitor monotherapy and that treatment with IPI-549 in combination with checkpoint inhibitors is able to overcome this resistance by reprogramming macrophages from the M2, pro-tumor phenotype to the M1, anti-tumor phenotype. Taken together, these preclinical data demonstrate that PI3K-gamma plays a key role in the immuno-suppressive tumor microenvironment, help to further elucidate the mechanism of action for IPI-549 and provide a strong rationale for the ongoing Phase 1 study of IPI-549.
First Quarter 2017 Financial Results

At March 31, 2017, Infinity had total cash, cash equivalents and available-for-sale securities of $75.4 million, compared to $92.1 million at December 31, 2016. Cash used for operating activities during the first quarter of 2017 included $5.2 million of payments related to the company’s 2016 restructuring activities.
Infinity did not record any revenue during the first quarter of 2017. Revenue for the first quarter of 2016 was $9.3 million, all of which related to Infinity’s previous collaboration agreement with AbbVie Inc.
Research and development (R&D) expense for the first quarter of 2017 was $4.0 million, compared to $39.2 million for the same period in 2016. The decrease in R&D expense was primarily related to a decrease in clinical development expenses for duvelisib in addition to the company’s 2016 restructuring activities.
General and administrative (G&A) expense was $6.4 million for the first quarter of 2017 compared to $10.8 million for the same period in 2016. The decrease in G&A expense was primarily due to the company’s 2016 restructuring activities.
Net loss for the first quarter of 2017 was $10.5 million, or a basic and diluted loss per common share of $0.21, compared to a net loss of $40.7 million, or a basic and diluted loss per common share of $0.82, for the first quarter of 2016.
Cash and Investments Outlook
Infinity’s 2017 financial outlook remains as follows:

Net loss: Infinity expects net loss for 2017 to range from $40 million to $50 million.
Cash and Investments: Infinity expects to end 2017 with a cash, cash equivalents and available-for-sale securities balance ranging from $40 million to $50 million.
Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities at March 31, 2017, will be adequate to satisfy the company’s capital needs into the first quarter of 2019. The company’s financial outlook excludes additional funding or business development activities.

About the IPI-549 and the Ongoing Phase 1 Study
IPI-549 is an investigational, orally administered immuno-oncology development candidate that selectively inhibits PI3K-gamma. In preclinical studies, IPI-549 reprograms macrophages from a pro-tumor to an anti-tumor phenotype and is able to overcome resistance to checkpoint inhibition.3,4 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially complementary approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

A Phase 1 study of IPI-549 in patients with advanced solid tumors is ongoing to explore the activity, safety, tolerability, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in patients with advanced solid tumors.5 The study includes monotherapy and combination dose-escalation phases, in addition to a monotherapy expansion cohort and combination expansion cohorts. The expansion cohorts evaluating IPI-549 plus Opdivo will include patients with non-small cell lung cancer (NSCLC), melanoma and squamous cell carcinoma of the head and neck (SCCHN). Patients enrolled in these combination expansion cohorts represent a difficult-to-treat population, as they must have demonstrated initial resistance or subsequently develop resistance to a PD-1 or PD-L1 therapy immediately prior to enrolling in the study. Overall, the study is expected to enroll approximately 175 patients.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.